Lymphangioleiomyomatosis is an ultra-rare disease mainly affecting women of childbearing age. The MILES trial showed the efficacy of sirolimus, an mTOR inhibitor, in stabilising lung function in patients with lymphangioleiomyomatosis. Drug toxicity and development of resistance are potential limitations of therapy with sirolimus. Nintedanib is a multikinase inhibitor that inhibits PDGFR, which is active in human and murine lymphangioleiomyomatosis lesions. We aimed to investigate the activity and safety of nintedanib in patients with lymphangioleiomyomatosis.

中文翻译：

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尼达尼布治疗淋巴管平滑肌瘤病患者：一项 2 期、开放标签、单臂研究


淋巴管平滑肌瘤病是一种极其罕见的疾病，主要影响育龄妇女。MILES 试验显示了西罗莫司（一种 mTOR 抑制剂）在稳定淋巴管平滑肌瘤病患者肺功能方面的疗效。药物毒性和耐药性的发展是西罗莫司治疗的潜在局限性。Nintedanib 是一种抑制 PDGFR 的多激酶抑制剂，PDGFR 在人和小鼠淋巴管平滑肌瘤病病变中具有活性。我们旨在研究尼达尼布在淋巴管平滑肌瘤病患者中的活性和安全性。